肌酸激酶-MM/原癌基因酪氨酸蛋白激酶受体作为杜氏肌肉萎缩症携带者的敏感指标

IF 4.6 2区 医学 Q1 NEUROSCIENCES
Molecular Neurobiology Pub Date : 2024-12-01 Epub Date: 2024-05-20 DOI:10.1007/s12035-024-04235-z
Zhilei Zhang, Dongyang Hong, Dingyuan Ma, Peiying Yang, Jingjing Zhang, Xin Wang, Yan Wang, Lulu Meng, Yanyun Wang, Yahong Li, Yun Sun, Tao Jiang, Zhengfeng Xu
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引用次数: 0

摘要

杜兴氏肌营养不良症(DMD)是一种致命的 X 连锁隐性遗传病,其特点是进行性肌肉萎缩,会导致患者在 20 多岁时因心肺并发症而过早死亡。而2.5%-19%的DMD携带者也会出现骨骼肌损伤或扩张型心肌病,如能尽早诊断,对产前诊断和自我保健的提前预警都很有意义。目前的 DMD 携带者筛查主要依靠检测血清肌酸激酶活性,只能覆盖 50-70% 的 DMD 携带者,这将导致许多假阴性结果,因此需要发现高效的生物标志物和简单的 DMD 携带者检测程序。在本文中,我们全面总结了所有与 DMD 相关的生物标志物,并根据其表达模式进行了分类。我们特别指出了以前从未在 DMD 携带者中报道过的新型 DMD 生物标志物,并对其进行了进一步研究,以探索其潜力。与 DMD 携带者体内单纯的肌酸激酶活性相比,肌酸激酶-MM 可将特异性从 73% 提高到 81%。我们的研究还发现了另一种有潜力的蛋白质:原癌基因酪氨酸蛋白激酶受体(RET)。当与肌酸激酶-MM(肌酸激酶-MM/RET比值)结合使用时,可显著提高检测血清中 DMD 携带者的特异性(从 81% 提高到 83%)和灵敏度(从 71.4% 提高到 93%)。此外,我们还成功设计出一种从干血斑中提取 RET 的有效方法。这一突破使我们能够利用干血斑同时检测肌酸激酶-MM和RET,而不影响检测率。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

Creatine Kinase-MM/Proto-oncogene Tyrosine-Protein Kinase Receptor as a Sensitive Indicator for Duchenne Muscular Dystrophy Carriers.

Creatine Kinase-MM/Proto-oncogene Tyrosine-Protein Kinase Receptor as a Sensitive Indicator for Duchenne Muscular Dystrophy Carriers.

Duchenne muscular dystrophy (DMD), a lethal X-linked recessive genetic disease, is characterized by progressive muscle wasting which will lead to premature death by cardiorespiratory complications in their late twenties. And 2.5-19% DMD carriers that also suffer from skeletal muscle damage or dilated cardiomyopathy when diagnosed as soon as possible is meaningful for prenatal diagnosis and advance warning for self-health. The current DMD carrier screening mainly relies on detecting serum creatine kinase activity, covering only 50-70% DMD carriers which will cause many false negatives and require the discovery of highly effective biomarker and simple detection procedure for DMD carriers. In this article, we have compiled a comprehensive summary of all documented biomarkers associated with DMD and categorized them based on their expression patterns. We specifically pinpointed novel DMD biomarkers, previously unreported in DMD carriers, and conducted further investigations to explore their potential. Compared to creatine kinase activity alone in DMD carriers, creatine kinase-MM can improve the specificity from 73 to 81%. And our investigation revealed another promising protein: proto-oncogene tyrosine-protein kinase receptor (RET). When combined with creatine kinase-MM (creatine kinase-MM/RET ratio), it significantly enhances the specificity (from 81 to 83%) and sensitivity (from 71.4 to 93%) of detecting DMD carriers in serum. Moreover, we successfully devised an efficient method for extracting RET from dried blood spots. This breakthrough allowed us to detect both creatine kinase-MM and RET using dried blood spots without compromising the detection rate.

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来源期刊
Molecular Neurobiology
Molecular Neurobiology 医学-神经科学
CiteScore
9.00
自引率
2.00%
发文量
480
审稿时长
1 months
期刊介绍: Molecular Neurobiology is an exciting journal for neuroscientists needing to stay in close touch with progress at the forefront of molecular brain research today. It is an especially important periodical for graduate students and "postdocs," specifically designed to synthesize and critically assess research trends for all neuroscientists hoping to stay active at the cutting edge of this dramatically developing area. This journal has proven to be crucial in departmental libraries, serving as essential reading for every committed neuroscientist who is striving to keep abreast of all rapid developments in a forefront field. Most recent significant advances in experimental and clinical neuroscience have been occurring at the molecular level. Until now, there has been no journal devoted to looking closely at this fragmented literature in a critical, coherent fashion. Each submission is thoroughly analyzed by scientists and clinicians internationally renowned for their special competence in the areas treated.
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